Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …

Retinitis Pigmentosa (RP) is a hereditary retinopathy that affects about 2.5 million people
worldwide. It is characterized with progressive loss of rods and cones and causes severe …

Adeno-associated virus (AAV) is a small, nonenveloped virus that was adapted 30 years
ago for use as a gene transfer vehicle. It is capable of transducing a wide range of species …

The emergence of genetic engineering at the beginning of the 1970′ s opened the era of
biomedical technologies, which aims to improve human health using genetic manipulation …

Inherited retinal dystrophies (IRDs) are a group of rare eye diseases caused by gene
mutations that result in the degradation of cone and rod photoreceptors or the retinal …

Leber congenital amaurosis (LCA) is a severe disorder resulting in visual impairment
usually starting in the first year of life. The most frequent genetic cause of LCA is an intronic …

Degenerative retinal diseases such as retinitis pigmentosa (RP) and age-related macular
degeneration (AMD) affect millions of people around the world and lead to irreversible vision …

Targeting the photosensitive ion channel channelrhodopsin‐2 (ChR2) to the retinal circuitry
downstream of photoreceptors holds promise in treating vision loss caused by retinal …

Inherited retinopathies (IR) are common untreatable blinding conditions. Most of them are
inherited as monogenic disorders, due to mutations in genes expressed in retinal …

Most inherited retinal dystrophies display progressive photoreceptor cell degeneration
leading to severe visual impairment. Optogenetic reactivation of retinal neurons mediated by …